FDA Approves PALYNZIQ for Adolescent PKU Treatment
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Feb 28 2026
0mins
Should l Buy BMRN?
Source: Newsfilter
- FDA Approval: BioMarin announced that the FDA has approved PALYNZIQ for adolescents aged 12 and older with PKU, marking an expansion of the drug's application in the adolescent population and is expected to improve their daily management.
- Clinical Trial Results: In the PEGASUS study, the PALYNZIQ group showed an average reduction of 473 μmol/L in blood phenylalanine (Phe) levels at Week 72, compared to a mere -19 μmol/L in the diet-only group, indicating a significant efficacy difference that could transform treatment for adolescent PKU patients.
- Increased Dietary Freedom: As the only enzyme substitution therapy, PALYNZIQ allows patients to manage Phe levels without dietary restrictions, helping adolescents alleviate the burdens of dietary limitations while facing academic and independence challenges.
- Safety and Side Effects: While PALYNZIQ's side effects include injection site reactions and hypersensitivity, most adverse reactions occurred during the induction phase in clinical trials, with a significant decrease in frequency during maintenance, indicating a favorable long-term safety profile.
Trade with 70% Backtested Accuracy
Stop guessing "Should I Buy BMRN?" and start using high-conviction signals backed by rigorous historical data.
Sign up today to access powerful investing tools and make smarter, data-driven decisions.
Analyst Views on BMRN
Wall Street analysts forecast BMRN stock price to rise
19 Analyst Rating
14 Buy
5 Hold
0 Sell
Moderate Buy
Current: 54.090
Low
60.00
Averages
87.35
High
120.00
Current: 54.090
Low
60.00
Averages
87.35
High
120.00
About BMRN
BioMarin Pharmaceutical Inc. is a global biotechnology company engaged in the development of genetic discovery into medicines that make an impact on the life of each patient. The Company has eight commercial therapies and a clinical and preclinical pipeline. Its commercial products include Vimizim (elosulfase alpha), Naglazyme (galsulfase), Palynziq (pegvaliase-pqpz), Brineura (cerliponase alfa), Aldurazyme (laronidase), Roctavian (valoctocogene roxaparvovec), Kuvan (sapropterin dihydrochloride), and Voxzogo (vosoritide). Vimizim is an enzyme replacement therapy for the treatment of MPS IVA, a lysosomal storage disorder. The Roctavian is an adeno-associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. Its key clinical programs include Vosoritide, BMN 333, BMN 390, BMN 351, BMN 349, and INZ-701. Its late-stage enzyme replacement therapy, INZ-701, is being developed for the treatment of ENPP1 deficiency.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
CNBC Launches Advocacy Platform for Rare Diseases
- Platform Launch: CNBC launched the CNBC Cures platform on January 8, aimed at giving a voice to the 30 million Americans living with rare diseases, connecting them with existing advocacy groups to drive attention and support from the medical community.
- Audience Engagement: The CNBC Cures newsletter has surpassed 20,000 subscribers, making it one of the fastest-growing newsletters on the network, indicating strong viewer interest and support for rare disease topics.
- Social Media Impact: Cures-related videos have garnered 1 million views across Facebook, TikTok, Instagram, and LinkedIn, demonstrating significant outreach and enhancing public awareness of rare diseases.
- Successful Summit: The inaugural CNBC Cures Summit attracted notable figures like Warren Buffett and sold out shortly after registration opened, with over 8,000 views for the livestream, reflecting heightened societal attention and support for rare disease issues.
See More
BioMarin Discontinues Voxzogo Trials Amid Safety Concerns
- Trial Discontinuation: BioMarin announced the discontinuation of dosing and enrollment in its phase 2 trials for Voxzogo in Turner syndrome, SHOX deficiency, and ACAN deficiency due to multiple slipped capital femoral epiphysis (SCFE) events, indicating potential safety concerns that could impact future drug development strategies.
- Safety Profile Maintained: Although no SCFE events were observed in BioMarin's other phase 2 trials and none in over 5,000 children treated with Voxzogo, the occurrence of these events may still undermine investor confidence in the company's products, potentially affecting stock performance.
- Continued Trials: BioMarin stated that the Canopy phase 2 trials for children with Noonan syndrome and idiopathic short stature (ISS) without ACAN deficiency will proceed as planned, suggesting that the company still sees potential in Voxzogo for certain indications, which may support future market performance.
- Stock Price Reaction: Following this announcement, BioMarin's shares fell 4.6% to $55.80 in premarket trading, reflecting the market's negative reaction to the trial discontinuation, which could impact its future financing and R&D capabilities.
See More
VOXZOGO Treatment Shows Significant Improvements in Children's Skeletal Growth
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm (p=0.0002) after four years, underscoring the critical importance of early intervention for skeletal growth.
- Proportionality Improvements: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and further improved to 0.53 (p<0.0001) by year four, demonstrating the drug's effectiveness in enhancing body proportionality.
- Global Clinical Evidence: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the drug's efficacy and safety.
- Long-Term Safety Studies: BioMarin will present findings on VOXZOGO's long-term safety and growth outcomes at the 2026 ACMG Annual Clinical Genetics Meeting, emphasizing its significant role in children's skeletal development and aiding communication between physicians and families.
See More
VOXZOGO Shows Significant Efficacy in Children
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm over four years (p=0.0002), underscoring the significant impact of early intervention on skeletal growth and reinforcing VOXZOGO's status as the only approved treatment.
- Proportionality Improvement: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and reached 0.53 (p<0.0001) by year four, indicating that early treatment enhances not only height but also overall body proportionality, which is crucial for health.
- Global Evidence Support: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the efficacy and safety of VOXZOGO.
- Clinical Research Findings: BioMarin will present these research results at the 2026 American College of Medical Genetics and Genomics Annual Meeting, emphasizing the importance of VOXZOGO in children's skeletal development and facilitating communication between families and healthcare providers.
See More
BridgeBio's Promising Biotech Pipeline Highlighted by William Blair
- Unique Market Position: Analyst John Boyle highlights BridgeBio's unique position with the accelerating launch of Attruby and near-term NDA submissions, setting the stage for late-2026/early-2027 launches in the rare genetic disease treatment market.
- Product Competitive Edge: Market surveys indicate that prescribers view both Attruby and Amvuttra as superior to Pfizer's Tafamidis, which has approximately $6 billion in annual sales, suggesting that BridgeBio's products are gaining traction in clinical applications.
- Sales Forecast and Risks: The analyst models U.S. sales of Attruby reaching $3.4 billion by 2035, while acknowledging potential generic entry risks in late 2028; however, Attruby's strong TTR stabilization and simple twice-daily dosing could justify premium pricing.
- Other Market Opportunities: BridgeBio also sees significant market opportunities with other rare disease drugs, particularly infigratinib for achondroplasia and encaleret for autosomal dominant hypocalcemia type 1, with the latter achieving its primary endpoint in the CALIBRATE Phase 3 study in October 2025, indicating strong future prospects.
See More
BioMarin Pharmaceutical Outlines Strategic Refresh and Market Outlook
- Strategic Update: BioMarin Pharmaceutical emphasized its strategic refresh at the TD Cowen conference, particularly through the Inozyme deal and the pending Amicus acquisition, aiming to expand enzyme therapy scale across approximately 80 countries, thereby enhancing its competitive position in the rare disease market.
- Revenue Outlook: Despite anticipating a roughly 3% revenue headwind in 2026 due to the removal of ROCTAVIAN and Kuvan revenue declines, BioMarin expects core enzyme and skeletal businesses to grow at mid-to-high single digits, targeting a 40% operating margin by 2026.
- Competitive Dynamics: BioMarin anticipates new FDA-approved competition for Voxzogo; however, management believes that patient switching will be gradual due to its five-year market lead and strong patient adherence, which will help maintain market share.
- Clinical Catalysts: BioMarin is advancing multiple clinical trials, including a Phase 3 study for hypochondroplasia and a pediatric Phase 3 study for BMN 401, which are expected to provide significant growth drivers for the company, particularly in the global rare disease treatment landscape.
See More
CNBC Launches Advocacy Platform for Rare Diseases
- Platform Launch: CNBC launched the CNBC Cures platform on January 8, aimed at giving a voice to the 30 million Americans living with rare diseases, connecting them with existing advocacy groups to drive attention and support from the medical community.
- Audience Engagement: The CNBC Cures newsletter has surpassed 20,000 subscribers, making it one of the fastest-growing newsletters on the network, indicating strong viewer interest and support for rare disease topics.
- Social Media Impact: Cures-related videos have garnered 1 million views across Facebook, TikTok, Instagram, and LinkedIn, demonstrating significant outreach and enhancing public awareness of rare diseases.
- Successful Summit: The inaugural CNBC Cures Summit attracted notable figures like Warren Buffett and sold out shortly after registration opened, with over 8,000 views for the livestream, reflecting heightened societal attention and support for rare disease issues.
See More
BioMarin Discontinues Voxzogo Trials Amid Safety Concerns
- Trial Discontinuation: BioMarin announced the discontinuation of dosing and enrollment in its phase 2 trials for Voxzogo in Turner syndrome, SHOX deficiency, and ACAN deficiency due to multiple slipped capital femoral epiphysis (SCFE) events, indicating potential safety concerns that could impact future drug development strategies.
- Safety Profile Maintained: Although no SCFE events were observed in BioMarin's other phase 2 trials and none in over 5,000 children treated with Voxzogo, the occurrence of these events may still undermine investor confidence in the company's products, potentially affecting stock performance.
- Continued Trials: BioMarin stated that the Canopy phase 2 trials for children with Noonan syndrome and idiopathic short stature (ISS) without ACAN deficiency will proceed as planned, suggesting that the company still sees potential in Voxzogo for certain indications, which may support future market performance.
- Stock Price Reaction: Following this announcement, BioMarin's shares fell 4.6% to $55.80 in premarket trading, reflecting the market's negative reaction to the trial discontinuation, which could impact its future financing and R&D capabilities.
See More
VOXZOGO Treatment Shows Significant Improvements in Children's Skeletal Growth
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm (p=0.0002) after four years, underscoring the critical importance of early intervention for skeletal growth.
- Proportionality Improvements: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and further improved to 0.53 (p<0.0001) by year four, demonstrating the drug's effectiveness in enhancing body proportionality.
- Global Clinical Evidence: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the drug's efficacy and safety.
- Long-Term Safety Studies: BioMarin will present findings on VOXZOGO's long-term safety and growth outcomes at the 2026 ACMG Annual Clinical Genetics Meeting, emphasizing its significant role in children's skeletal development and aiding communication between physicians and families.
See More
VOXZOGO Shows Significant Efficacy in Children
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm over four years (p=0.0002), underscoring the significant impact of early intervention on skeletal growth and reinforcing VOXZOGO's status as the only approved treatment.
- Proportionality Improvement: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and reached 0.53 (p<0.0001) by year four, indicating that early treatment enhances not only height but also overall body proportionality, which is crucial for health.
- Global Evidence Support: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the efficacy and safety of VOXZOGO.
- Clinical Research Findings: BioMarin will present these research results at the 2026 American College of Medical Genetics and Genomics Annual Meeting, emphasizing the importance of VOXZOGO in children's skeletal development and facilitating communication between families and healthcare providers.
See More
BridgeBio's Promising Biotech Pipeline Highlighted by William Blair
- Unique Market Position: Analyst John Boyle highlights BridgeBio's unique position with the accelerating launch of Attruby and near-term NDA submissions, setting the stage for late-2026/early-2027 launches in the rare genetic disease treatment market.
- Product Competitive Edge: Market surveys indicate that prescribers view both Attruby and Amvuttra as superior to Pfizer's Tafamidis, which has approximately $6 billion in annual sales, suggesting that BridgeBio's products are gaining traction in clinical applications.
- Sales Forecast and Risks: The analyst models U.S. sales of Attruby reaching $3.4 billion by 2035, while acknowledging potential generic entry risks in late 2028; however, Attruby's strong TTR stabilization and simple twice-daily dosing could justify premium pricing.
- Other Market Opportunities: BridgeBio also sees significant market opportunities with other rare disease drugs, particularly infigratinib for achondroplasia and encaleret for autosomal dominant hypocalcemia type 1, with the latter achieving its primary endpoint in the CALIBRATE Phase 3 study in October 2025, indicating strong future prospects.
See More
BioMarin Pharmaceutical Outlines Strategic Refresh and Market Outlook
- Strategic Update: BioMarin Pharmaceutical emphasized its strategic refresh at the TD Cowen conference, particularly through the Inozyme deal and the pending Amicus acquisition, aiming to expand enzyme therapy scale across approximately 80 countries, thereby enhancing its competitive position in the rare disease market.
- Revenue Outlook: Despite anticipating a roughly 3% revenue headwind in 2026 due to the removal of ROCTAVIAN and Kuvan revenue declines, BioMarin expects core enzyme and skeletal businesses to grow at mid-to-high single digits, targeting a 40% operating margin by 2026.
- Competitive Dynamics: BioMarin anticipates new FDA-approved competition for Voxzogo; however, management believes that patient switching will be gradual due to its five-year market lead and strong patient adherence, which will help maintain market share.
- Clinical Catalysts: BioMarin is advancing multiple clinical trials, including a Phase 3 study for hypochondroplasia and a pediatric Phase 3 study for BMN 401, which are expected to provide significant growth drivers for the company, particularly in the global rare disease treatment landscape.
See More
